A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD (POSITANO)

Camarus

Status and phase: Active, not recruiting; Phase 3; Phase 2

Conditions: Polycystic Liver Disease

Treatments: Drug: Placebo; Drug: CAM2029

Study type: Interventional

Funder types: Industry

Identifiers

NCT05281328

2021-003764-27 (EudraCT Number)

HS-20-677

Details and patient eligibility

About

The purpose of the trial is to compare the effectiveness and safety of 2 treatment regimens of CAM2029 (given weekly or every 2 weeks) to placebo in participants with symptomatic PLD, either isolated as in autosomal dominant PLD (ADPLD) or associated with autosomal dominant polycystic kidney disease (ADPKD).

In the Treatment Period of the trial, participants will be allocated at random to 1 of the 3 treatment arms in a 1:1:1 ratio. After completing the Treatment Period (53 weeks) participants may proceed to a 120-week open-label extension part of the trial and then only receive the same CAM2029 treatment.

The active ingredient in CAM2029, octreotide, is administered as a subcutaneous depot using Camurus' FluidCrystal® technology.

Enrollment: 71 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female patient, ≥18 years at screening
• Diagnosis of PLD (associated with ADPKD or isolated as in ADPLD) as defined by htTLV ≥1800 mL/m at screening
• Presence of at least 1 of the following PLD-related symptoms within 2 weeks before screening: bloating, fullness in abdomen, lack of appetite, feeling full quickly after beginning to eat, acid reflux, nausea, rib cage pain or pressure, pain in side, abdominal pain, back pain, shortness of breath after physical exertion, limited in mobility, concern about abdomen getting larger, dissatisfied by the size of abdomen
• Not a candidate for, or not willing to undergo, surgical intervention for hepatic cysts during the trial

Exclusion criteria

• Surgical intervention for PLD within 3 months before screening
• Treatment with a somatostatin analogue (SSA) within 3 months before screening
• Non-responsive to previous treatment of PLD with an SSA as per the Investigator's assessment
• Systematic cholelithiasis within 3 months before screening or previous medical history of cholelithiasis induced by SSAs unless treated with cholecystectomy
• Presence of extrahepatic cysts that, in the Investigator's opinion, may prevent the patient from safely participating in the trial
• Severe kidney disease, as defined by eGFR <30 mL/min/1.73^m2
• Severe liver disease defined as liver cirrhosis of Child-Pugh class C
• Any other current or prior medical condition that may interfere with the conduct of the trial or the evaluation of its results in the opinion of the Investigator

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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71 participants in 3 patient groups, including a placebo group

CAM2029 once weekly

Experimental group

Description:

0.5 mL CAM2029 10 mg, subcutaneous (SC) injection, once weekly

Treatment:

Drug: CAM2029

CAM2029 once every 2 weeks

Experimental group

Description:

0.5 mL CAM2029 10 mg, SC injection, every 2 weeks and 0.5 mL placebo, SC injection, once every 2 weeks (alternating with CAM2029 dosing)

Treatment:

Drug: CAM2029

Drug: Placebo

Placebo

Placebo Comparator group

Description:

0.5 mL placebo, SC injection, once weekly

Treatment:

Drug: Placebo